Real-world data and evidence in health technology assessment: When are they complementary, substitutes, or the only sources of data compared to clinical trials? - 25/01/23

Doi : 10.1016/j.therap.2022.11.001

Gérard de Pouvourville ^a, Xavier Armoiry ^b,^⁎ , Aurélie Lavorel ^c, Pascal Bilbault ^d, Philippe Maugendre ^e, Lionel Bensimon ^f,^{^{1
The participants of the “Health and economy” round table of the Giens Workhsops 2022.}}, Dan Beziz ^g,^{^{1
The participants of the “Health and economy” round table of the Giens Workhsops 2022.}}, Patrick Blin ^h,^{^{1
The participants of the “Health and economy” round table of the Giens Workhsops 2022.}}, Isabelle Borget ^i,^{^{1
The participants of the “Health and economy” round table of the Giens Workhsops 2022.}}, Stéphane Bouée ^j,^{^{1
The participants of the “Health and economy” round table of the Giens Workhsops 2022.}}, Cécile Collignon ^k,^{^{1
The participants of the “Health and economy” round table of the Giens Workhsops 2022.}}, Benoît Dervaux ^l,^{^{1
The participants of the “Health and economy” round table of the Giens Workhsops 2022.}}, Isabelle Durand-Zaleski ^m,^{^{1
The participants of the “Health and economy” round table of the Giens Workhsops 2022.}}, Marc Julien ^n,^{^{1
The participants of the “Health and economy” round table of the Giens Workhsops 2022.}}, Lucie de Léotoing ^o,^{^{1
The participants of the “Health and economy” round table of the Giens Workhsops 2022.}}, Laureen Majed ^p,^{^{1
The participants of the “Health and economy” round table of the Giens Workhsops 2022.}}, Nicolas Martelli ^q,^{^{1
The participants of the “Health and economy” round table of the Giens Workhsops 2022.}}, Thomas Séjourné ^e,^{^{1
The participants of the “Health and economy” round table of the Giens Workhsops 2022.}}, Marie Viprey ^r,^s,^{^{1
The participants of the “Health and economy” round table of the Giens Workhsops 2022.}}
^a ESSEC Business School, 75011 Paris, France
^b Université Claude Bernard Lyon 1, Institut des sciences pharmaceutiques et biologiques (ISPB)/UMR CNRS 5510 MATEIS/Hôpital Edouard Herriot, service pharmaceutique, 690008 Lyon, France
^c SNITEM, 92400 Courbevoie, France
^d LYSARC, centre hospitalier Lyon sud, 69495 Pierre Bénite, France
^e Sanofi, 75008 Paris, France
^f MSD, 92800 Puteaux, France
^g Novartis, 92300 Levallois Perret, France
^h Université de Bordeaux, 33076 Bordeaux, France
ⁱ Institut Gustave Roussy, 92000 Villejuif, France
^j CEMKA, 92340 Bourg-la-Reine, France
^k Medtronic, 75014 Paris, France
^l CHRU Lille, 59037 Lille, France
^m Université de Paris, CRESS–APHP, 75004 Paris, France
ⁿ Diabeloop, 78230 Grenoble, France
^o W.L. Gore & Associés, 75012 Paris, France
^p Health Data Hub, Paris 75000 France
^q AP-HP–Université Paris-Saclay, 91400 Orsay, France
^r Hospices civils de Lyon, Health Data Department, Lyon, France
^s Research on Healthcare Performance RESHAPE, INSERM U1290, Université Claude Bernard Lyon 1, 69000 Lyon, France

^⁎Corresponding author: Université Claude Bernard Lyon 1, Institut des Sciences Pharmaceutiques et Biologiques (ISPB)/UMR CNRS 5510 MATEIS/Hôpital Edouard Herriot, Service Pharmaceutique, 8, avenue Rockefeller, 69008 Lyon, France.Université Claude Bernard Lyon 1, Institut des Sciences Pharmaceutiques et Biologiques (ISPB)/UMR CNRS 5510 MATEIS/Hôpital Edouard Herriot, Service Pharmaceutique8, avenue RockefellerLyon69008France

Summary

Within the life-cycle assessment of health technologies, real-world data (RWD) have until now been of secondary importance to clinical trial data. The availability of massive, better quality RWD, particularly with the emergence of connected devices, the improvement of methods for characterizing populations, make it possible to have a better insight into the effects of treatment, sometimes on a national scale the importance of RWD is likely to progress in the eyes of health technology assessors, going from being traditionally complementary to possibly replacing clinical trial data. This is the fundamental question that the round table, involving experts from the academic and/or hospital, institutional, and industrial worlds, set out to answer. This work served first to establish the current role of RWD in health technology assessment, by distinguishing the main purposes of RWD, the timing of the evaluation in relation to the life cycle of the technology, and then according to the party commissioning or receiving the outcomes of RWD-based studies. Secondly, the round table proposed six general recommendations for more intensive and decisive use of RWD in the assessment and decision-making process.

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Keywords : Real-world data, Health technology assessment, Decision making

Plan

Abbreviations

Introduction and scope of the Round Table

Methodology

Working method

Framework

Current role of real-world data in health technology assessment

Real-world data in context assessment

Real-world data in effectiveness and safety assessment

Real-world data in economic analysis

Real-world data in organizational impact assessment

Recommendations

Recommendation 1: Facilitate the linking of data sources

Recommendation 2: Have sufficient permanent means and rules shared by all parties to ensure and reinforce the quality of the RWD collected and their accessibility.

Recommendation 3: In terms of the main SNDS database, encourage the collaborative development, validation and sharing of algorithms for identifying diseases/events of interest/endpoints/unit costs.

Recommendation 4: In the context of organizational impact, support multicentre or multiservice pathway studies, choose measurement indicators from the literature and weight them, and develop the use of multicriteria decision support tools (details in paragraphe “real-world data in organizational impact assessment”).

Recommendation 5: Develop training (initial and continuing) and information for stakeholders on data sources, their possible uses and limitations, and strengthen knowledge and training on methodological standards relating to RWD.

Recommendation 6: Support and stimulate the development of real-world data collection, such as quality of life and resource consumption data, to improve the validity of cost-effectiveness studies

Disclosure of interest

☆	The articles, analyses and proposals resulting from the New Giens Workshops are those of the authors and do not prejudge the positions of their organisations.

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Vol 78 - N° 1

P. 81-94 - janvier 2023 Retour au numéro

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Données et preuves en vie réelle dans l’évaluation des technologies de santé : dans quels cas sont-elles complémentaires, substitutives, ou les seules sources de données par rapport aux essais cliniques ?
Gérard de Pouvourville, Xavier Armoiry, Aurélie Lavorel, Pascal Bilbault, Philippe Maugendre, Lionel Bensimon, Dan Beziz, Patrick Blin, Isabelle Borget, Stéphane Bouée, Cécile Collignon, Benoît Dervaux, Isabelle Durand-Zaleski, Marc Julien, Lucie de Léotoing, Laureen Majed, Nicolas Martelli, Thomas Séjourné, Marie Viprey

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Real-world data and evidence in health technology assessment: When are they complementary, substitutes, or the only sources of data compared to clinical trials? - 25/01/23

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